Recombinant adeno-associated virus (rAAV) vectors have become essential in gene therapy for delivering therapeutic genes effectively. However, the challenge of separating empty capsids from genome-containing particles remains a significant hurdle, impacting product purity, safety, and overall efficacy. Traditional methods, such as density gradient ultracentrifugation, have been widely used but present scalability and consistency issues. Recent developments in anion exchange chromatography (AEX) using weak organic acids provide an innovative solution, allowing efficient separation and enrichment of genome-containing capsids without the limitations of gradient elution. This paper discusses the successful application and scalability of AEX for rAAV purification, offering a promising pathway for large-scale gene therapy manufacturing.
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October 25, 2024
Displacement Chromatography for Enrichment of rAAV Genome-Containing Capsids using Weak Organic Acid
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Repeatability and Intermediate Precision of a Genome Integrity Assay for rAAV by Long-Read Sequencing
Truncated recombinant AAV genomes can compromise transgene expression and the therapeutic efficacy1 of gene therapy vectors, making genome integrity a critical quality attribute, highlighting the need for robust analytical methods. Long‑read...
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ddPCR-Based Quantification and Fragment Analysis of Residual E1A in rAAV Products: Method Validation and Correlation with Total Host Cell DNA
Abstract Gene therapy using adeno-associated virus (AAV) vectors has emerged as a promising strategy, offering new hope for patients with genetic disorders. However, during rAAV manufacturing, residual host-cell DNA (hcDNA) and plasmid DNA from the...
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June 01, 2026
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Multivariate Approach to Small-Scale Model Qualification in Upstream Adeno-Associated Virus Production
Abstract AAV gene therapy manufacturing requires scalable processes and reliable small-scale models (SSMs) to support process characterization and regulatory expectations. Ensuring that SSMs accurately represent large‑scale operations is essential...
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June 01, 2026